by Sajeve Thomas, MD, Oncologist
Biliary tract cancer (BTC), a rare and aggressive form of cancer affecting the bile ducts, gallbladder, and liver, has historically been challenging to treat. However, there is hope on the horizon with the recent FDA approval of pembrolizumab (Keytruda, Merck) in combination with chemotherapy. This groundbreaking development offers new possibilities for patients and their healthcare providers, potentially improving outcomes for those with locally advanced unresectable or metastatic BTC.
Understanding Pembrolizumab and Its Role in BTC Treatment
Pembrolizumab is a medication classified as an immune checkpoint inhibitor. It works by enhancing the body’s immune response against cancer cells. In this case, pembrolizumab has been approved for use in combination with gemcitabine and cisplatin, two chemotherapy drugs, for patients with locally advanced unresectable or metastatic BTC.
Key Clinical Trial Results
The FDA’s approval of pembrolizumab with chemotherapy for BTC is based on promising results from the KEYNOTE-966 clinical trial. This trial enrolled 1,069 patients who had not received prior systemic therapy for advanced BTC. The patients were randomized into two groups: one received pembrolizumab in combination with gemcitabine and cisplatin, and the other received a placebo with the same chemotherapy regimen.
The primary measure of efficacy in this trial was overall survival (OS). The results were remarkable, with the pembrolizumab combination demonstrating a statistically significant improvement in OS compared to the placebo combination. The median OS was 12.7 months for the pembrolizumab group and 10.9 months for the placebo group.
Potential Side Effects and Dosing
Like all medications, pembrolizumab can have side effects. In this trial, 55% of patients experienced adverse reactions that led to the interruption of pembrolizumab. The most common side effects included decreased neutrophil count, decreased platelet count, anemia, decreased white blood cell count, pyrexia, fatigue, cholangitis, increased ALT, increased AST, and biliary obstruction.
The recommended dose of pembrolizumab is 200 mg every 3 weeks or 400 mg every 6 weeks until disease progression or unacceptable toxicity. It is important to note that pembrolizumab should be administered before chemotherapy when given on the same day.
Project Orbis and Faster Approvals
The FDA’s approval of pembrolizumab for BTC was conducted under Project Orbis, an initiative that facilitates concurrent submission and review of oncology drugs among international partners. This approach expedites the approval process and enables patients to access innovative treatments sooner.
Reporting Adverse Events
For healthcare providers and caregivers, it is crucial to report any serious adverse events associated with the use of pembrolizumab or any other medicine to the FDA’s MedWatch Reporting System. Timely reporting is vital in ensuring patient safety and ongoing research into the medication’s efficacy.
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The FDA’s approval of pembrolizumab in combination with chemotherapy for biliary tract cancer is a significant step forward in the treatment of this challenging disease and adds to the current evidence that chemoimmunotherapy is an incremental small step forward to improving outcomes in patients dealing with metastatic BTC. For patients, caregivers, and healthcare providers, this development offers renewed hope and a potential lifeline for those affected by BTC. It is a testament to the progress in oncology research and the dedication of scientists, healthcare professionals, and regulatory agencies in the fight against cancer.
As we continue to advance in the field of oncology, it is essential to stay informed about the latest breakthroughs and treatment options. The FDA’s commitment to expediting approvals and Project Orbis’s international collaboration demonstrates the collective effort to improve the lives of cancer patients worldwide. Stay tuned for further updates and advancements in the field of oncology, and remember that your healthcare team is here to support you throughout your journey.
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About the author
Dr. Sajeve Thomas is a distinguished medical professional and a compassionate guide in the field of oncology. With over a decade of dedicated experience as a board-certified medical oncologist/internal medicine specialist, Dr. Thomas has become a trusted expert in the treatment of melanoma, sarcoma, and gastrointestinal conditions. Currently practicing at the renowned Orlando Health Cancer Institute, he brings a wealth of expertise to the complex and challenging world of oncology.